Paroxysmal Nocturnal Hemoglobinuria - Market Insight, Epidemiology And Market Forecast - 2032

DelveInsight's 'Paroxysmal Nocturnal Hemoglobinuria(PNH) - Market Insights, Epidemiology, and Market Forecast-2032' report delivers an in-depth understanding of Paroxysmal Nocturnal Hemoglobinuria historical and forecasted epidemiology as well as the Paroxysmal Nocturnal Hemoglobinuria market trends in the United States, the EU4 (Germany, France, Italy, Spain), the UK and Japan.

The PNH market report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted 7MM PNH market size. The report also covers PNH procedure, SWOT analysis, and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.

Geography Covered:

• The United States
• EU4 (Germany, France, Italy, Spain) and the UK
• Japan

Study Period: 2019-2032.

Paroxysmal Nocturnal Hemoglobinuria Understanding

PNH Overview

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired disorder of the pluripotent hematopoietic stem cell; therefore, it can affect erythrocytes, leukocytes, thrombocytes, and probably some endothelial cells. These hematopoietic stem cells have acquired a somatic mutation in an X-linked gene: the phosphatidylinositol glycan class A (PIG-A). This gene synthesizes the glycosylphosphatidylinositol (GPI) anchor, which is necessary to attach some proteins to the cell membrane.

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Clinical Forms of PNH

A clinical polymorphism of PNH has been described. Three forms are identified: classical PNH, PNH associated with aplastic anemia (AA), and subclinical PNH; however, it is important to understand that these entities are three clinical manifestations of a single disease.

Classical form

The classical form of PNH affects young people preferentially. They suffer from chronic intravascular hemolytic anemia due to a continuous state of complement activation, but brisk periods of hemolysis may result from complement activation due to infection, surgery, strenuous activity, and alcohol intake.in adults. The risk of bleeding correlates to the severity of thrombocytopenia. Patients may present without symptoms, minimal bleeding, or serious hemorrhage (e.g., mucosal, intracranial, gastrointestinal, genitourinary). Older patients, patients on antiplatelet therapy, and patients with comorbid conditions may have severe bleeding manifestations.

PNH associated with aplastic anemia

When PNH is associated with AA, most patients express only a small PNH clone (<10%). Physicians could find clinical and/or biological evidence of hemolysis associated with two or three cytopenias (Hb <10 g/dL, neutrophils <1,000/μL, platelets <80,000/μL). In general, bone marrow failure dominates the clinical picture.

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PNH Epidemiology

The epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by Total Diagnosed Prevalence of PNH, Gender-specific Prevalence of PNH scenario in the 7MM covering the United States, the EU4 countries (Germany, France, Italy, and Spain), the United Kingdom and Japan from 2019 to 2032.

Key Findings

• In 2021, the total cases of PNH in the 7MM countries were 12,358 and these cases are anticipated to increase during the study period. The US accounted for 6,043 cases of PNH in 2021.
• Among the EU4 countries, Germany had the highest number of cases of PNH in 2021, i.e., 1,355 cases, followed by the France which had 851 cases in 2021. On the other hand, Italy had the lowest number of cases of PNH, i.e. 250 cases in 2021.
• The United Kingdom accounted for 2,569 cases of PNH in 2021.
• In case of gender-specific prevalence of PNH, in 2021 females accounted for 3,148 cases as compared to 2,894 cases in males in the United States.

PNH Drug Chapters

PNH Emerging Drugs

Iptacopan: Novartis

Iptacopan, or LNP023, is a first-in-class, orally administered, potent, and highly selective factor B inhibitor of the alternative complement pathway. LNP023 is currently in clinical development for PNH and several renal conditions with complement system involvement, including IgA nephropathy, complement 3 glomerulopathies (C3G), atypical hemolytic uremic syndrome, and membranous nephropathy. In PNH, LNP023 acts upstream of the C5 terminal pathway, preventing intravascular hemolysis and extravascular hemolysis. In doing so, LNP023 may have a therapeutic advantage over the current standard of care by targeting the underlying pathophysiology.

Product details in the report…

Crovalimab: Hoffmann-La Roche

Crovalimab is also known as RG6107, and SKY59 is a humanized complement inhibitor C5 monoclonal antibody (anti-C5 recycling antibody) discovered by Chugai using recycling antibody technology. SKY59 is designed to target C5, a key component of the complement system, and is expected to control complement activity. It inhibits complement activation by blocking the cleavage of c5 to c5a and c5b, which is the cause of PNH. Roche is codeveloping SKY59 with Chugai Pharmaceuticals.

Products detail in the report…

Danicopan: Alexion Pharmaceuticals

ALXN2040 (also known as danicopan) is an investigational, first-in-class, oral proximal factor D inhibitor developed by Achillion, a wholly owned subsidiary of Alexion. Factor D (FD), a serine protease, catalyzes the cleavage of complement factor B into Ba and Bb, which allows for the formation of the AP C3 convertase. By inhibiting FD, danicopan, blocks C3 convertase formation, the control point for AP activation, and the amplification of all pathways. This leads to the inhibition of C3 cleavage, C3 fragment deposition, terminal pathway activation, and MAC formation.

Product details in the report…

PNH Market Outlook

PNH is a rare hematological disorder characterized by episodes of hemolysis, thrombosis, and bone marrow failure and sometimes progresses to myelodysplastic syndromes. It has fascinated hematologists for more than a century due to its diverse manifestations and intricate pathophysiology. PNH can sink in 2 ways: one is a classical form called anemia due to hemolysis because of the defect of surface molecules; another is aplastic anemia. In some patients, both of these forms can sink into one another. PNH is typically diagnosed in a nonacute setting.

The disease-modifying therapeutic strategies for PNH are complement inhibition therapy and bone marrow transplantation. Eculizumab is a licensed therapy for PNH, and its efficacy has relegated bone marrow transplantation to second-line therapy for hemolytic PNH in countries where the drug is available. Bone marrow transplantation might be an option if eculizumab is unavailable, and it is a reasonable therapeutic strategy in patients with PNH and severe bone marrow failure. Adjunctive therapies (for example, immunosuppression) could be prescribed to patients with PNH who also have bone marrow failure to ameliorate the latter. However, these adjunctive treatments are not specific to PNH nor have consistent effects on the expansion or reduction of PNH clones. The only curative strategy for PNH is allogeneic stem cell transplantation, but this procedure continues to carry a considerable risk of mortality and is considered for patients who have severe bone marrow failure.

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Key Findings

• The market size of PNH in the 7MM was observed to be USD 1123 million in 2021.
• In 2021, the US has the largest PNH market size of USD 703 million among the 7MM countries.
• Among the EU4 countries, Germany had the highest market size for PNH in 2021, i.e., USD 90 million. The lowest market size was estimated in Italy with USD 17 million in 2021.
• In Japan, the market size for PNH was observed to be USD 63 million in 2021.

The United States Market Outlook

This section provides the total PNH market size and market size by therapies in the United States.

EU4 and the UK Market Outlook

The total PNH market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom are provided in this section.

Japan Market Outlook

The total PNH market size and market size by therapies in Japan are provided.

PNH Drugs Uptake

This section focuses on the rate of uptake of the potential drugs recently launched in the PNH market or expected to get launched in the market during the study period 2019-2032. The analysis covers the PNH market uptake by drugs; patient uptake by therapies; and sales of each drug.

This helps in understanding the drugs with the most rapid uptake, and reasons behind the maximal use of new drugs, and allows the comparison of the drugs based on market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

PNH Development Activities

The report provides insights into different therapeutic candidates in Phase II and Phase III stage. It also analyzes key players involved in developing targeted therapeutics.

Pipeline Development Activities

The report covers detailed information on collaborations, acquisitions, merger, licensing, and patent details for PNH emerging therapies.

Reimbursement Scenario in PNH

Approaching reimbursement proactively can have a positive impact both during the late stages of product development and well after product launch. In the report, we consider reimbursement to identify economically attractive indications and market opportunities. When working with finite resources, the ability to select the markets with the fewest reimbursement barriers can be a critical business and price strategy.

Competitive Intelligence Analysis

We perform competitive market Intelligence analysis of the PNH market by using various competitive intelligence tools that include-SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report:

• The report covers a descriptive overview of PNH, explaining its procedure, types, indications, and currently available therapies.
• Comprehensive insight has been provided into PNH epidemiology and treatment.
• Additionally, an all-inclusive account of both the current and emerging therapies for PNH is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.
• A detailed review of the PNH market; historical and forecasted is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies, by understanding trends shaping and driving the 7MM PNH market.

Report Highlights:

• The robust pipeline with novel MOA and oral ROA, increasing prevalence, and effectiveness of drugs will positively drive the PNH market.
• The companies and academics are working to assess challenges and seek opportunities that could influence PNH R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition using PNH.
• Major players are involved in developing therapies for PNH. The launch of emerging therapies will significantly impact the PNH market.
• Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

PNH Report Insights

• Patient Population
• Therapeutic Approaches
• PNH Pipeline Analysis
• PNH Market Size and Trends
• Market Opportunities
• Impact of upcoming Therapies

PNH Report Key Strengths

• 11-Year Forecast
• 7MM Coverage
• PNH Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed Market
• Drugs Uptake

PNH Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• SWOT analysis

Key Questions

Market Insights:

• What was the PNH market share (%) distribution in 2019 and how it would look like in 2032?
• What would be the PNH total market size as well as market size by therapies across the 7MM during the forecast period (2022-2032)?
• What are the key findings pertaining to the market across the 7MM and which country will have the largest PNH market size during the forecast period (2022-2032)?
• At what CAGR, the PNH market is expected to grow at the 7MM level during the forecast period (2022-2032)?
• What would be the PNH market outlook across the 7MM during the forecast period (2022-2032)?
• What would be the PNH market growth till 2032 and what will be the resultant market size in the year 2032?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights:

• What are the risk, burdens, and unmet needs of PNH?
• What is the historical PNH patient pool in the United States, EU4 (Germany, France, Italy, Spain), the UK and Japan?
• What would be the forecasted patient pool of PNH at the 7MM level?
• What will be the growth opportunities across the 7MM with respect to the patient population pertaining to PNH?
• Out of the above-mentioned countries, which country would have the highest cases of PNH during the forecast period (2022-2032)?
• At what CAGR are these cases expected to grow across the 7MM during the forecast period (2022-2032)?

Current Treatment Scenario, Marketed Drugs, and Emerging Therapies:

• What are the current treatment practices in PNH along with the approved therapy?
• What are the current treatment guidelines for PNH?
• What are the PNH marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety, and efficacy, etc.?
• How many companies are developing therapies for PNH?
• How many emerging therapies are in the mid-stage and late stages of development for PNH?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the PNH therapies?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for PNH and their status?
• What are the key designations that have been granted for the emerging therapies for PNH?
• What are the 7MM historical and forecasted market of PNH?

Reasons to buy:

• The report will help in developing business strategies by understanding trends shaping and driving PNH.
• To understand the future market competition in the PNH market and an insightful review of the market.
• Organize sales and marketing efforts by identifying the best opportunities for PNH in the US, EU4 (Germany, Spain, Italy, France), the United Kingdom, and Japan.
• Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.
• Organize sales and marketing efforts by identifying the best opportunities for the PNH market.
• To understand the future market competition in the PNH market.